Capping is a required protection in 5′ of the mRNA that is necessary for biological activity and stability into the cells. There are already several methods of capping using mCap, ARCA or enzymatic methods. Unfortunately, they are limited in efficiency (such as mCap and ARCA), and can be costly (such as the enzymatic methods). Trilink Biotechnologies, the modified nucleic acid experts, have developped a new, far more efficient technology called CleanCap available in Europe via tebu-bio.

A few years ago, Ayal Hendel et al (doi:10.1038/nbt.3290) published results revealing that chemical alterations to sgRNA enhance gene editing in primary cells. To demonstrate this, Matthew H Porteus’s team chose the targeted genes CCR5, HBB and IL2RG respectively involved in anti-HIV clinical trials, cell anaemia and thalassemia, and severe combined immunodeficiency. More recently, the same team tested several modified CAS9 mRNA. You can find the practical results on this poster introduced at the ASGC.

Today, I’d like to invite you to take a look at a highly efficient and useful kit, which brings together all the required components you need in a complete system for culturing and transfecting human pluripotent stem cells for gene editing.